CRISPR-Cas12a allowing for simultaneous editing of multiple genes in mammalian cells
Problem: CRISPR-based gene editing technique has revolutionized the field of molecular biology. While CRISPR-Cas9-based methods are effectively used for single-gene knockout, multiplex gene editing remains a major challenge. Unlike Cas9, Cas12a can be used for multigene editing from a single RNA transcript but demonstrates low gene editing efficiency in mammalian cells.
Solution: Dr. Shi and his team have optimized the Cas12a gene editing system to allow for highly efficient knockout of multiple genes (up to 20) in mammalian cells. The system allows for multiple applications including (i) stratification of process of multiple gene editing for research projects, gene therapy, or cell therapy, (ii) combinatorial genetic screening in various cell types, (iii) fine-tuning expression of a gene of interest by controlling expression of multiple regulatory areas, and (iv) discovering gene interactions across diverse disease types.
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Docket # 20-9242